EP3471780A4 - Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept - Google Patents

Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept Download PDF

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Publication number
EP3471780A4
EP3471780A4 EP17814223.8A EP17814223A EP3471780A4 EP 3471780 A4 EP3471780 A4 EP 3471780A4 EP 17814223 A EP17814223 A EP 17814223A EP 3471780 A4 EP3471780 A4 EP 3471780A4
Authority
EP
European Patent Office
Prior art keywords
aflibercept
amd
treatment
aav2 variant
aav2
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
EP17814223.8A
Other languages
German (de)
English (en)
Other versions
EP3471780A2 (fr
EP3471780B1 (fr
Inventor
Mark Blumenkranz
Mehdi Gasmi
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Adverum Biotechnologies Inc
Original Assignee
Adverum Biotechnologies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Priority to RS20201517A priority Critical patent/RS61311B1/sr
Priority to SI201730553T priority patent/SI3471780T1/sl
Priority to EP20196657.9A priority patent/EP3795181A1/fr
Publication of EP3471780A4 publication Critical patent/EP3471780A4/fr
Publication of EP3471780A2 publication Critical patent/EP3471780A2/fr
Application granted granted Critical
Publication of EP3471780B1 publication Critical patent/EP3471780B1/fr
Priority to HRP20201842TT priority patent/HRP20201842T1/hr
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/178—Syringes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Dispersion Chemistry (AREA)
  • Mycology (AREA)
  • Hematology (AREA)
EP17814223.8A 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept Active EP3471780B1 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
RS20201517A RS61311B1 (sr) 2016-06-16 2017-06-16 Lečenje amd korišćenjem varijante aav2 sa afliberceptom
SI201730553T SI3471780T1 (sl) 2016-06-16 2017-06-16 Zdravljenje SDM-ja z uporabo različice AAV2 z afliberceptom
EP20196657.9A EP3795181A1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept
HRP20201842TT HRP20201842T1 (hr) 2016-06-16 2020-11-19 Liječenje amd korištenjem aav2 varijante s afliberceptom

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662351234P 2016-06-16 2016-06-16
PCT/US2017/038003 WO2017218974A2 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept

Related Child Applications (2)

Application Number Title Priority Date Filing Date
EP20196657.9A Division EP3795181A1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept
EP20196657.9A Division-Into EP3795181A1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept

Publications (3)

Publication Number Publication Date
EP3471780A4 true EP3471780A4 (fr) 2019-04-24
EP3471780A2 EP3471780A2 (fr) 2019-04-24
EP3471780B1 EP3471780B1 (fr) 2020-10-28

Family

ID=60663653

Family Applications (2)

Application Number Title Priority Date Filing Date
EP17814223.8A Active EP3471780B1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept
EP20196657.9A Pending EP3795181A1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept

Family Applications After (1)

Application Number Title Priority Date Filing Date
EP20196657.9A Pending EP3795181A1 (fr) 2016-06-16 2017-06-16 Traitement de la dmla à l'aide d'un variant d'aav2 et d'aflibercept

Country Status (22)

Country Link
US (2) US20190151409A1 (fr)
EP (2) EP3471780B1 (fr)
JP (4) JP6814822B2 (fr)
KR (2) KR20210021113A (fr)
CN (2) CN109641065A (fr)
AU (2) AU2017286673B2 (fr)
BR (1) BR112018076124A2 (fr)
CA (1) CA3027737A1 (fr)
CY (1) CY1123697T1 (fr)
DK (1) DK3471780T3 (fr)
ES (1) ES2840059T3 (fr)
HR (1) HRP20201842T1 (fr)
HU (1) HUE051953T2 (fr)
IL (3) IL279919B1 (fr)
LT (1) LT3471780T (fr)
MX (2) MX2018015512A (fr)
PT (1) PT3471780T (fr)
RS (1) RS61311B1 (fr)
SG (1) SG11201811232XA (fr)
SI (1) SI3471780T1 (fr)
WO (1) WO2017218974A2 (fr)
ZA (1) ZA201808538B (fr)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201810150UA (en) 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
CN107405507B (zh) 2015-03-02 2022-05-03 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
EP3528785A4 (fr) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. Capsides d'aav modifiées et leurs utilisations
EP3596213A4 (fr) 2017-03-17 2021-02-17 Adverum Biotechnologies, Inc. Compositions et procédés d'amplification d'expression génique
CN107537026A (zh) * 2017-08-31 2018-01-05 中山大学中山眼科中心 Vegf‑b的应用
CN107661492A (zh) * 2017-08-31 2018-02-06 中山大学中山眼科中心 Vegf‑b的新用途
AU2018350990A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
US11766489B2 (en) * 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP2021506861A (ja) 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のａａｖ媒介送達
EP3773677A1 (fr) * 2018-04-12 2021-02-17 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Compositions anti-âge et procédés d'utilisation
US20200297869A1 (en) * 2019-03-04 2020-09-24 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
MX2022002961A (es) * 2019-09-11 2022-04-06 Adverum Biotechnologies Inc Metodos para tratar enfermedades neovasculares oculares usando variantes de aav2 que codifican para aflibercept.
KR20220062352A (ko) * 2019-09-11 2022-05-16 애드베룸 바이오테크놀로지스, 인코포레이티드 아플리베르셉트를 코딩하는 aav2 변이체를 사용하여 안구 신생혈관 질환을 치료하는 방법
US11400039B2 (en) * 2020-01-16 2022-08-02 James W. Hill Changing eye color by gene transduction
CN113952474A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CA3186830A1 (fr) * 2020-07-21 2022-01-27 Zhongdong SHI Composition et methode pour traiter des maladies oculaires
CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CN113817775B (zh) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 修饰的阿柏西普、组合物、方法及其在基因治疗中的应用
JP2021049374A (ja) * 2020-12-09 2021-04-01 株式会社三洋物産 遊技機
EP4329721A1 (fr) * 2021-04-27 2024-03-06 Adverum Biotechnologies, Inc. Méthodes de traitement de maladies oculaires au moyen de variantes d'aav2 codant pour l'aflibercept
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof

Citations (2)

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Publication number Priority date Publication date Assignee Title
US20140371438A1 (en) * 2012-05-15 2014-12-18 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1
WO2016141078A1 (fr) * 2015-03-02 2016-09-09 Avalanche Biotechnologies, Inc. Compositions et procédés d'administration intravitréenne de polynucléotides à des cônes rétiniens

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RS62795B1 (sr) * 2011-04-22 2022-02-28 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
ES2753135T3 (es) * 2012-05-07 2020-04-07 Allergan Inc Método de tratamiento de DMAE en pacientes resistentes a terapia anti-VEGF
EP3019243A4 (fr) * 2013-07-12 2017-03-15 Ophthotech Corporation Procédés pour traiter ou prévenir des états ophtalmologiques
AU2014337135B2 (en) * 2013-10-18 2019-09-19 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a VEGF antagonist and an anti-CTLA-4 antibody

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
US20140371438A1 (en) * 2012-05-15 2014-12-18 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1
WO2016141078A1 (fr) * 2015-03-02 2016-09-09 Avalanche Biotechnologies, Inc. Compositions et procédés d'administration intravitréenne de polynucléotides à des cônes rétiniens

Non-Patent Citations (2)

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Title
DENIZ DALKARA ET AL: "In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous", SCIENCE TRANSLATIONAL MEDICINE, 12 June 2013 (2013-06-12), United States, pages 1 - 11, XP055533378, Retrieved from the Internet <URL:http://stm.sciencemag.org/content/scitransmed/5/189/189ra76.full.pdf> DOI: 10.1126/scitranslmed.3005708 *
PETER PECHAN ET AL: "Gene Therapies for Neovascular Age-Related Macular Degeneration", COLD SPRING HARBOR PERSPECTIVES IN MEDICINE, vol. 5, no. 7, 18 December 2014 (2014-12-18), pages a017335, XP055556414, DOI: 10.1101/cshperspect.a017335 *

Also Published As

Publication number Publication date
MX2022004786A (es) 2022-05-16
ES2840059T3 (es) 2021-07-06
IL263686B (en) 2021-02-28
RS61311B1 (sr) 2021-02-26
IL279919A (en) 2021-03-01
JP6814822B2 (ja) 2021-01-20
CN109641065A (zh) 2019-04-16
KR20190037228A (ko) 2019-04-05
JP2022137244A (ja) 2022-09-21
CY1123697T1 (el) 2022-03-24
PT3471780T (pt) 2020-12-17
MX2018015512A (es) 2019-04-24
BR112018076124A2 (pt) 2019-03-26
AU2017286673A1 (en) 2019-01-31
DK3471780T3 (da) 2020-11-23
AU2017286673B2 (en) 2021-06-24
AU2021225178A1 (en) 2021-09-30
JP2024113193A (ja) 2024-08-21
CN117531025A (zh) 2024-02-09
HRP20201842T1 (hr) 2021-04-16
WO2017218974A3 (fr) 2018-01-25
ZA201808538B (en) 2021-04-28
IL314568A (en) 2024-09-01
LT3471780T (lt) 2021-01-25
KR20210021113A (ko) 2021-02-24
CA3027737A1 (fr) 2017-12-21
EP3471780A2 (fr) 2019-04-24
SG11201811232XA (en) 2019-01-30
IL263686A (en) 2019-01-31
IL279919B1 (en) 2024-09-01
SI3471780T1 (sl) 2021-03-31
US20190151409A1 (en) 2019-05-23
EP3795181A1 (fr) 2021-03-24
WO2017218974A2 (fr) 2017-12-21
HUE051953T2 (hu) 2021-04-28
EP3471780B1 (fr) 2020-10-28
JP2019521990A (ja) 2019-08-08
JP2020203941A (ja) 2020-12-24
KR102218265B1 (ko) 2021-02-25
US20220265740A1 (en) 2022-08-25

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